TY - EJOU AU - Shimomura, Tatsuya AU - Murakami, Masaya AU - Kasai, Kanako AU - Imai, Yu AU - Inaba, Yuzo AU - Kimura, Takahiro TI - Administrating Everolimus against neuroendocrine differentiated prostate cancer, a preliminary experience T2 - Canadian Journal of Urology PY - VL - IS - SN - 1488-5581 AB - Background: Neuroendocrine prostate cancer (NEPC) is a deadly condition, with a median overall survival of less than one year after diagnosis. Although clinical trials are ongoing, an established treatment strategy for NEPC has not yet been developed. A recent clinical trial, RADIANT-4, showed that treatment with everolimus significantly improved survival in patients with progressive neuroendocrine tumors of the lung or gastrointestinal tract. This study evaluated the efficacy of everolimus against neuroendocrine differentiated prostate cancer as an initial investigation. Methods: Five patients with neuroendocrine differentiated prostate cancer were treated with everolimus (10 mg daily). In our study, we examined the responses and changes over time of circulating neuroendocrine tumor markers, including neuron-specific enolase (NSE) and pro-gastrin-releasing peptide (pro-GRP). We also evaluated survival outcomes such as castration-resistant prostate cancer (CRPC)-free survival, overall survival (OS), and cancer-specific survival (CSS). Results: The median follow-up duration was 32 months. A decline in circulating neuroendocrine tumor markers was observed in all cases. The median decline rate of NSE was −0.417, and that of pro-GRP was −0.647 (p = 0.841). The median CRPC-free survival was 10 months (range: 3–NA). Median CSS and OS were both 32 months (range: 8–NA). Grade 3 adverse events (AEs) included hyperglycemia and pneumonitis. Grade 1 AEs included stomatitis (2 cases), paronychia, and pneumonitis. Conclusions: Everolimus demonstrated reductions in circulating neuroendocrine markers in this exploratory cohort of prostate adenocarcinoma with neuroendocrine differentiation; however, these findings are descriptive and do not establish treatment efficacy. KW - everolimus; neuroendocrine differentiation; prostate cancer; neuroendocrine tumor markers; neuron-specific enolase; pro-gastrin-releasing peptide DO - 10.32604/cju.2026.076841